So since drug pricing and FDA regulations are so much in the news, it would seem like the perfect time for a small company to game the system for big profits, right? That’s apparently what Marathon Pharmaceuticals believes. They just got approval for deflazacort, a steroid, as a treatment for Duchenne Muscular Dystrophy. Duchenne is a terrible disease, of course, and for bringing a drug onto the market for a rare pediatric disease, Marathon gets a Priority Review Voucher (as the law calls for – a reward for companies entering these areas). They also get 7 years of market exclusivity for bringing an orphan drug not previously available in the US under FDA approval – that’s provided by law as well, as an incentive.
So what’s not to like? Well, this drug has been around since the early 1990s. Marathon most certainly did not invent it. Nor did they think of applying it to DMD patients – the biggest clinical trial of the drug for that indication was done over twenty years ago, by someone else. DMD patients in the US were already taking the (unapproved) drug by importing it from Canada. Marathon just dug through the data again and ran a trial in 29 patients themselves, from what I can see. I should note that this is not any sort of cure, nor does it address the underlying pathology of the disease. The steroid treatment makes muscle strength in DMD patients stronger – barely. But even for that benefit, US patients will now have to get it from Marathon at something like 50 to 100 times the former price. This is exactly the same business plan as Catalyst Pharmaceuticals and several others, and the only reason that it’s viable is because perverse incentives by the FDA make it completely legal.
Update: for more on Marathon’s costs, see here.
So while I defend the FDA’s function of making it tough on new drugs (making them prove safety and efficacy), I cannot stand how loose they are with old generic compounds. The agency hands out extremely valuable rewards like lollipops in these cases – a priority review voucher can be sold for hundreds of millions of dollars, for example. They are wildly overvaluing the worth of a “Now FDA Approved!” designation in the cases of things that have been used around the world for many years.
And they’re also allowing the likes of Marathon to make the rest of the drug industry look like greedy sociopaths. Marathon, Catalyst, T*ring and all the rest of the people who are pulling these tricks have the word “Pharmaceuticals” in their name, but they are not drug companies. They discover nothing. They do no research. They take virtually no risks. They exist only to play legal games and watch the money roll in.
Marathon, for their part, is a member of PhRMA, which just adds luster to John LaMattina’s call for the large research companies to give up on the trade organization entirely (he’s been critical of them for some time now). No one should be comfortable standing next to something like this. As for the FDA, the agency probably can’t change this on its own, though, even if it wants to – Congress has to act to give them the authority to deny market exclusivity or priority review vouchers under some conditions. Either that, or we should rethink these incentives entirely, because they are (clearly) too easy to exploit for fast bucks.